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EVALUATION STUDIES
JOURNAL ARTICLE
Increased complications and morbidity in children with hemophagocytic lymphohistiocytosis undergoing hematopoietic stem cell transplantation.
Clinical Transplantation 2013 March
BACKGROUND: Hematopoietic stem cell transplantation (HSCT) is the only curative option for patients with primary hemophagocytic lymphohistiocytosis (HLH) and for patients with secondary HLH who fail to respond to therapy.
METHODS: Retrospective study of HSCT for HLH with focus on complications and outcome.
RESULTS: Eighteen children (10 males), with a median age of 1.2 yr (5 months-16 yr), received HSCT for HLH. Fourteen children had primary HLH. Four children underwent transplant while not in remission. Sixteen received myeloablative and two received reduced intensity conditioning regimen. A high incidence of complications was found: 13 (72%) children had 22 episodes of culture-proven infections; seven (38%) had hepatic veno-occlusive disease; nine (50%) developed respiratory complications; and nine (50%) required intensive care unit admission. Eight children had acute graft-versus-host disease (GVHD), and three developed chronic GVHD. Three patients died from multi-organ failure before day +100, and another patient died from pulmonary hemorrhage after day 100. Three patients failed to engraft (two developed recurrent HLH and died from complications after a second HSCT). Three of four children not in remission at the time of transplantation died. Actuarial survival at three yr was 61%.
CONCLUSION: HSCT for HLH carries significant risks with high infection, organ dysfunction, and ICU admissions rates.
METHODS: Retrospective study of HSCT for HLH with focus on complications and outcome.
RESULTS: Eighteen children (10 males), with a median age of 1.2 yr (5 months-16 yr), received HSCT for HLH. Fourteen children had primary HLH. Four children underwent transplant while not in remission. Sixteen received myeloablative and two received reduced intensity conditioning regimen. A high incidence of complications was found: 13 (72%) children had 22 episodes of culture-proven infections; seven (38%) had hepatic veno-occlusive disease; nine (50%) developed respiratory complications; and nine (50%) required intensive care unit admission. Eight children had acute graft-versus-host disease (GVHD), and three developed chronic GVHD. Three patients died from multi-organ failure before day +100, and another patient died from pulmonary hemorrhage after day 100. Three patients failed to engraft (two developed recurrent HLH and died from complications after a second HSCT). Three of four children not in remission at the time of transplantation died. Actuarial survival at three yr was 61%.
CONCLUSION: HSCT for HLH carries significant risks with high infection, organ dysfunction, and ICU admissions rates.
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